Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis
NCT ID: NCT06449001
Last Updated: 2025-12-23
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
6 participants
INTERVENTIONAL
2025-08-11
2028-03-10
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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NON_RANDOMIZED
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Danicopan
Participants will receive a 12-week weight-based open-label treatment period and up to 1 year open-label long term extension period.
Danicopan
Participants will receive danicopan on a weight-based dosing regimen.
Interventions
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Danicopan
Participants will receive danicopan on a weight-based dosing regimen.
Eligibility Criteria
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Inclusion Criteria
* CS-EVH defined by: Anemia: Hgb ≤ 11.0 g/dL, and absolute reticulocyte count ≥ 100 × 109/L
* Treated with ravulizumab or eculizumab for at least 12 weeks immediately preceding Day 1, the dose received should be stable during this period, and there should be no anticipated changes in dosage or interval during the first 12 weeks of this study.
* all participants must be vaccinated against meningococcal infection from serogroups A, C, W, and Y and serogroup B within 3 years prior to, or at least 14 days prior to Day 1
* vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae
Exclusion Criteria
* ANC \< 500/μL.
* Clinically significant laboratory abnormalities related to liver function, including:
* ALT \> 2 × ULN or ALT \> 3 × ULN for participants with documented liver iron overload defined by serum ferritin values ≥ 500 ng/mL.
* Direct bilirubin \> 2 × ULN, unless, in the Investigator's opinion, is due to hemolysis or Gilbert's syndrome based on medical history.
* Current evidence of biliary cholestasis.
* Known aplastic anemia or other bone marrow failure that requires HSCT or other therapies, including anti-thymocyte globulin and immunosuppressants unless the dosage of immunosuppressant has been stable for at least 12 weeks before Day 1 and is expected to remain stable through Week 12.
* History of a major organ transplant (eg, heart, lung, kidney, liver) or HSCT.
* Known or suspected complement deficiency.
* Active bacterial or viral infection, a body temperature \> 38°C on 2 consecutive daily measures, evidence of other infection, or history of any febrile illness within 14 days prior to first study intervention administration.
12 Years
17 Years
ALL
No
Sponsors
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AstraZeneca
INDUSTRY
Alexion Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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Research Site
Saskatoon, Saskatchewan, Canada
Research Site
Paris, , France
Research Site
Leeds, , United Kingdom
Research Site
London, , United Kingdom
Countries
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Central Contacts
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Alexion Pharmaceuticals, Inc. (Sponsor)
Role: CONTACT
Phone: 1-855-752-2356
Email: [email protected]
Other Identifiers
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ALXN2040-PNH-302
Identifier Type: OTHER
Identifier Source: secondary_id
D7332C00006
Identifier Type: -
Identifier Source: org_study_id